RENSSELAER N.Y., August 30, 2017 – Age-related macular degeneration (AMD), a progressive degenerative disease of the retina, is the leading cause of blindness in the elderly. Patients with AMD gradually lose central vision, severely compromising the ability of a person to perform everyday tasks, including reading. AMD affects more than 10 million people in the US and is projected to increase to 196 million in 2020 worldwide. Scientists at the Neural Stem Cell Institute, including former NSCI researcher Christine Zhao and NSCI Principal Investigator Dr. Nathan Boles, have now published a breakthrough technique in the video journal Jove.

In AMD, the retinal pigment epithelium (RPE), a pigmented layer of cells lying beneath and supporting the retina, degenerates. AMD occurs in two forms, dry and wet. While there are therapies for the wet form of AMD, there are no approved therapies for the dry form of AMD. The advent of induced pluripotent stem cell (iPSCs), and their ability to generate any cell type in the body, has enabled the creation of cell culture models of disease that enable rapid drug screening for potential therapeutic agents.

Zhao and her team created a unique surgical technique to deliver cells to the sub-retical space of rat eyes: Subretinal injection has been widely applied in preclinical studies of stem cell replacement therapy for age-related macular degeneration. In this visualized article, we describe a less risky, reproducible and precisely modified subretinal injection technique via the trans-scleral approach to deliver cells into rat eyes.

A Molecular Biology class from Averill Park High School visited the labs at NSCI. These twenty-one 9th and 10th graders were able to learn from researchers, explore the lab and experience a range of scientific wet lab activities first hand.

Dr. Sally Temple answers questions from APHS students.

Leading the workshop, NSCI’s Dr. Thomas Kiehl describes the class teacher, Averill Park’s Greg Panzanaro, known as “Dr.P” as super motivated “He has tons of energy and is involved in a lot of different educational development opportunities. He regularly engages in workshops with other science teachers and seeks to involve students in those meetings as well. He’s continually developing new materials. He keeps students engaged. The amount of energy he brings to the classroom is amazing!”

Because Dr.P’s class handles some fairly advanced material Dr. Kiehl thought they would get a lot out of a visit to NSCI. They would gain some exposure to the lab environment and to some real career paths in science. He and NSCI lab manager Susan Goderie discussed what would best match the students’ existing skill set and simultaneously stretch their knowledge. Dr. Kiehl notes a challenge, “The biggest thing was coming up with something that fit within the time frame and would show them something significant.” Interestingly, Dr. Kiehl shares, the main activity that the students did is something that is routinely one of the first skills taught to NSCI college level interns.

Susan Goderie, NSCI Research Manager, explains the experiment for the students.

The tour began with an overview from Institute Scientific Director, Dr. Sally Temple. Then, Dr. Nathan Boles and graduate student, Amber Frye spoke. Dr. Kiehl explains, “One of the things I wanted to emphasize was the background of the scientists. How did they get interested in science? What decisions did they make? It’s usually a pretty personal journey for someone. ” Dr. Boles discussed stems cells—what they do and what you can do with them–and Ms. Frye spoke about the eye and retinal research being done at NSCI.

From there, students were invited into the lab bays and dressed in official personal protective equipment or “PPE” and performed an experiment involving human cells. They learned from Dr. David Butler about how the human cell lines they were working with were derived from patients. These specific cells were derived from NSCI work with Tauopathies. David explained that the cells were from patients who have the Tau disorder. Cells from these patients were reverted back to an induced pluripotent stem cell (a type of cell that

can turn into many other cell types) and then differentiated into astrocytes, which are the brain cells that help ‘clean up’ the brain when there is a trauma or injury. One of the cell lines retained the Tau mutation and the other cell line was one where the mutation had been corrected. Some of the cells had been swollen just as would happen naturally in response to an insult. After applying a stain to the cells the students used a fluorescent microscope to see the cells’ internal structure: the cytoskeleton and nuclei. They were able to compare the normal cells to the swollen cells.

The group was also introduced to the Neuracell staff and learned about the equipment and functioning of a core facility. They toured StemCultures, the for-profit business that makes and sells cell reagents. They even had a chance to look at live heart cells beating and learn about electrophysiological activity. Also packed into the tour, they were exposed to microscopy, witnessing the equipment and process of photographing cells. After their visit all of the images that were captured from their experiment were sent back with them along with videos of the beating heart cells.

Four astrocyte cultures stained by students from Averill Park high school. The blue stain labels the nuclei while the red stain labels F-actin, a component of the cytoskeleton of these cells.

Looking forward Dr. Kiehl sees the workshop series expanding and working with Dr. P. to refine future students’ experience. He feels that it’s important for young people to see the actual activities of working scientists and to hear about the science from individuals who are developing it. The students can gain the perspective of a real-life laboratory and a more sophisticated understanding of the field.

Dr. P and his students were thrilled by the 2½-hour workshop. He exclaims:

“This is such a remarkable opportunity that your company has offered as a real world experience.”

Dr. David Butler explains his work investigating tauopathies and the patient-derived cells the students got to work with.

Stressing the unique educational opportunity, Dr. Kiehl shares, “To hear first hand that this is a real career path. Students might not understand how to get from the lab they are doing in high school to this as a career. To have the opportunity to relate to the people who are doing this, they may be able to visualize the possibility. The advantage we have here is that we have several entities: the non-profit research arm, for-profit reagent company and a core facility that provides services to other laboratories. Students have the opportunity to learn some of the various career paths that follow in science.”

Dr. Temple was asked to participate on the show as a stem cell expert and the current President of the International Society for Stem Cell Research (ISSCR). The show focused on the problem of clinics peddling unproven stem cell therapies direct to consumers, typically for large sums of money. Hundreds of such clinics exist in the US alone. Patients are lured into these bogus and potentially harmful “treatments” with false promises. Dr. Temple was asked to speak about the difference between these unproven therapies, and authentic, responsible stem cell therapy development, which has the promise to bring forth treatments that are both safe and effective. Patients were advised to visit the website A Closer Look at Stem Cells to learn more about what to ask when considering a stem cell therapy.

She was also interviewed about her appearance on the show by the Times Union. Read the article here.

Dr. Temple was asked to participate on the show as a stem cell expert and the current President of the International Society for Stem Cell Research (ISSCR). The show focuses on the problem of clinics peddling unproven stem cell therapies direct to consumers, typically for large sums of money. Hundreds of such clinics exist in the US alone. Patients are lured into these bogus and potentially harmful “treatments” with false promises. Dr. Temple was asked to speak about the difference between these unproven therapies, and authentic, responsible stem cell therapy development, which has the promise to bring forth treatments that are both safe and effective. Patients were advised to visit the website A Closer Look at Stem Cells to learn more about what to ask when considering a stem cell therapy.

RENSSELAER N.Y., February 10, 2017 – Neural Stem Cell Institute Scientific Director, Dr. Sally Temple is one of the first 30 recipients of the new R35 Research Program Award, which is designed to encourage creative research by enhancing funding stability. Awards were announced by the National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health (NIH). NINDS-supported investigators who secure an R35 will have their research funded for a period of five years with the potential to have funding extended for up to an additional three years.

In her investigations, “Defining characteristics of cortical progenitor cells over time in mouse and human”, Dr. Temple and her research team are employing stem cell technology to study how the brain forms, and how neural stem cells can be activated to counteract developmental and neurodegenerative disorders.” It’s a great honor to receive this unique award, which enables us to take on significant challenges that impact brain health” said Temple.

The NINDS (http://www.ninds.nih.gov) is the nation’s leading funder of research on the brain and nervous system. “NINDS created the R35 program to improve the value of the research it funds by enabling proven investigators to pursue long-range, innovative research instead of continually writing and submitting grant applications,” said Walter Koroshetz, M.D., the NINDS director. Applications for the R35 award were reviewed according to NIH peer review standards, which include an assessment of investigators’ track records and the significance and relevance of their proposed research programs. The 30 awardees include principal investigators at a variety of career stages, striving to better understand the causes of neurodegenerative disease, how the brain develops, the molecular and cellular changes that give rise to memory, the origins of pain, and how to promote neural repair.

Dr. Temple is the 2008 recipient of the MacArthur Fellowship and is the current president of the International Society for Stem Cell Research (ISSCR).

About NSCI: NSCI, the first independent, non-profit stem cell research institute in the United States, is a unique research organization that produces leading stem cell science with the goal of developing novel therapies for diseases of the retina, brain and spinal cord. NSCI has a team of over 30 researchers focused on bringing impactful therapies to patients.

Our Unique Organization

The Neural Stem Cell Institute (NSCI) is dedicated to developing regenerative stem cell therapies for various diseases of the central nervous system (CNS): the brain, spinal cord and retina, regardless of the cause of injury. Led by MacArthur “Genius” Award winner Dr. Sally Temple who helped discover and define nervous system stem cells, NSCI is the first independent, non-profit stem cell research institute in the USA.